AZP-3601 SAD and MAD Study in Healthy Subjects and Patients With Hypoparathyroidism

NCT ID: NCT05239221

Last Updated: 2025-09-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 132 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-09-07
• Study Completion Date: 2022-08-23

Brief Summary

This study is investigating the safety, tolerability, pharmacodynamics and pharmacokinetics of AZP-3601 following single and repeated administration in both healthy volunteers and patients with chronic hypoparathyroidism (cHP)

The protocol includes 3 parts:

• Part A: first-in-human single ascending dose (SAD) study in healthy volunteers
• Part B: multiple ascending dose (MAD) study with 2 weeks of treatment in healthy volunteers
• Part C: open-label MAD study with a total treatment duration of 3 months in patients with cHP.

Conditions

Chronic Hypoparathyroidism

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

AZP-3601

subcutaneous (sc) administration once daily

Group Type: EXPERIMENTAL

AZP-3601

Intervention Type: DRUG

Lyophilized powder of AZP-3601 to be reconstituted with water for injection before injection

Placebo (Parts A and B)

subcutaneous (sc) administration once daily

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Saline solution visually matching active medication

Interventions

AZP-3601

Lyophilized powder of AZP-3601 to be reconstituted with water for injection before injection

Intervention Type: DRUG

Placebo

Saline solution visually matching active medication

Intervention Type: DRUG

Other Intervention Names

eneboparatide

Eligibility Criteria

Inclusion Criteria

• Part A: healthy male volunteers aged 18 to 60 years old inclusive with a body mass index of 19 to 28 kg/m2
• Part B: healthy male and female volunteers (non-child bearing potential) aged 18 to 60 years inclusive with a Body mass index of 19 to 28 kg/m2
• Part C:

1. Male and female patients aged 18 to 75 years inclusive

2. History of cHP for ≥12 months at the time of screening with documentation of two measurements of serum calcium and parathyroid hormone (PTH).

3. Requirement for therapy with calcitriol ≥0.25 μg per day or alphacalcidol ≥0.50 μg per day (both are active vitamin D supplements), and requirement for supplemental oral calcium treatment ≥1000 mg per day over and above normal dietary calcium intake at baseline assessments.

Exclusion Criteria

• Parts A and B:

1. Clinically significant abnormal lab values, as judged by the investigator

2. Using tobacco products with 3 months prior to first drug administration

3. History of alcohol abuse or drug addiction

• Part C:

1. Known history of autosomal-dominant hypocalcemia (ADH resulting from gain-of-function calcium-sensing receptor [CaSR] or GNA11 mutations) or pseudohypoparathyroidism (impaired responsiveness to PTH)

2. Any current disease that might affect calcium metabolism or calcium phosphate homeostasis other than HP

3. Use of medications such as loop and thiazide diuretics, raloxifene hydrochloride, lithium, methotrexate, cardiac glycosides (e.g., digoxin or digitoxin) or systemic corticosteroids within 4 weeks prior to start of treatment.

4. Previous treatment with PTH-like drugs, including PTH(1-84), PTH(1-34), or abaloparatide, within 3 months prior to screening.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Amolyt Pharma

INDUSTRY

Sponsor Role: collaborator

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Amolyt Pharma Investigational Site Hungary

Budapest, , Hungary

PRA-EDS

Groningen, , Netherlands

Countries

Hungary; Netherlands

References

Ovize M, Allas S, Culler MD, Milano S, Ouldrouis T, Sumeray M, van de Wetering de Rooij J, Mannstadt M. Phase 1 clinical trial of eneboparatide, a novel PTH receptor 1 agonist. Endocr Connect. 2025 Jun 19;14(6):e240464. doi: 10.1530/EC-24-0464. Print 2025 Jun 1.

Reference Type: DERIVED

PMID: 40423237 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

AZP-3601-CLI-001

Identifier Type: -

Identifier Source: org_study_id